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The Partnerships in Clinical Trials Blog focuses optimization intelligence, regulatory trends and globalization strategies for both as sponsors and CROs. It is supported by a number of industry events:

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Tuesday, March 3, 2015

Despite the tremendous contributions medical devices have
made to medicine, historically physicians viewed devices with suspicion and
were wary of machine-produced information. Today, the U.S. medical device
industry is the largest in the world with a market size of approximately $110
billion and is expected to reach $133 billion by 2016.1 Despite the industry
expansion, many of the challenges medical devices faced in the past continue to
affect modern-day markets. While new technologies are developed at a rapid
pace, providers and payers may remain slow to adopt them.

Sufficient data demonstrating effectiveness, safety and
value from multiple perspectives is key to the adoption of new devices. For
health care providers, devices should be practical and meet recognized clinical
needs. For payers, a device’s combined clinical and economic value must be
clear before coverage is provided. Addressing the needs and concerns of payers
and end users can be challenging.

Seeing Value Through
the Payer’s Eyes

In the U.S., when we refer to “payers,” we traditionally
mean government payers, such as the Centers for Medicare & Medicaid
Services (CMS) or private payers such as the Blue Cross Blue Shield
Association. More and more, health care facilities (e.g., hospitals) directly
purchase medical devices without receiving reimbursement from traditional
payers. In these cases, facilities act as payers, too.

The expanding role of health care facilities as payers is
spurred by health care reform, the creation of Accountable Care Organizations
(ACOs) and a shift in emphasis from volume-based to value-based reimbursement.
For innovative medical device adoption and success, identifying appropriate
payers and market strategies revolving around those payers’ perceptions is
crucial.

It is critical for medical device manufacturers to remember
that traditional health care payers are not impressed with the safety and
efficacy data collected in clinical trials for regulatory approval; payers seek
evidence of real-world effectiveness as compared to currently available
treatments. Furthermore, while often not explicitly stated, cost considerations
are implicit in payer decisions to cover innovative technologies.

Payers and providers are also moving together toward
value-based reimbursement where payers offer financial incentives to providers
for improvements in population health. However, because value-based
reimbursement is not yet the norm, manufacturers must design studies to collect
sufficient short- and long-term data to demonstrate device safety, real-world
effectiveness and economic impact from the traditional volume-based payer
perspective as well as from the myriad value-based reimbursement perspectives
still evolving.

Finally, when conducting research it is important to
remember the role of hospitals and other health care facilities as direct
purchasers of some technologies. Hospitals also want information on how an
innovative technology will affect their patients’ health as well as their
bottom line.

Paving the Road Ahead

Demonstration of long-term clinical and economic value from
the payers’ perspective is necessary to gain coverage and adoption. It is
crucial for every step of the development process to take place with the
technology’s value story in mind. The right research and a well-formulated
market access strategy will save substantial time and money and lead to earlier
market adoption and widespread usage of medical devices and other technologies.

Partnerships and Applied Clinical Trials chose this webcast to highlight a very important topic in the clinical research enterprise—the needs to understand and address the clinical investigative sites needs. While April Lewis and Ying Jiang at IMS Health provided information on data used for feasibility, qualification, negotiation (contracts and Fair Market Value), they also urged sponsors and CROs to share this data with the investigators to start a supportive dialogue to address their needs.

Why would this matter? Well, Tufts CSDD recently reported the decline of investigators—experienced investigators listing their increased burdens, as well as the novice investigators who conduct one trial and leave. Neither is good for maintaining clinical research in the long run noted Green. Green also agreed with Lewis that data be the foundation to very important discussions that lead to true collaborations.

Tuesday, February 17, 2015

Citeline have released their 2015 Annual Review regarding the trends in pharmaceutical R&D. Ian Lloyd, the reviewer for the past 25 years gives insights into new trends and the implications of them. In this blog I shall summarise the main points that are discussed, however for more detailed analysis the whole whitepaper has to be read.

One of the main focuses of the paper is total pipeline size and this year the figure has once again risen. The increase of 8.8% from 2014 takes the number of drugs in the development pipeline up to 12,300. The study then broke down the pipeline into different clinical stages and found that the phase with the highest number of drugs was in the preclinical phase. 6061 drugs were found to be in the pipeline in 2015 in the preclinical phase compared to 5484 in 2014. The mostly sought after data is from the three stages of clinical development. For the three stages there were increases across the board; for phase one, two and three there were increases of 4.9%, 7.0% and 8.6% respectively.

The report goes on to break down the pipeline figures into which companies are doing the best in terms of pipeline size. The top 25 is given but I have picked out the top 3:

1. GlaxoSmithKline – 258 drugs in the pipeline (2015)

2. Novartis – 245 drugs in the pipeline (2015)

3. Roche – 234 drugs in the pipeline (2015)

GlaxoSmithKline remains top of the pile despite slipping from 261 drugs in 2014 with the two next companies trading positions from 2014. A notable point in the table is that UK based AstraZeneca took 4th spot after fighting off a huge takeover bid from US based Pfizer who could only make 7th. However, how long can AZ fight off the Pfizer onslaught? More takeovers are likely because as it stands there are 3,286 companies with active pipelines. 56% of these companies are very small and Lloyd believes they could easily be gobbled up. The majority of the companies are found to be from the United States; 47% of R&D companies have headquarters in America, only losing 1% to the UK from 2014.

In terms of what therapeutic areas are dominating the pipelines, anticancer still leads the way with biotechnology a close second, despite not strictly being a therapeutic group. The worst performer was in the neurological area which only had a 4.5% increase from 2014. In terms of individual diseases, cancer types lead the way with 15 out of the top 25 being forms of cancer. The highest number of active drugs which are non-cancer are for rheumatoid arthritis and type 2 diabetes that came 4th and 5th respectively in the study.

Of all the drugs in the pipeline, the vast majority came from a synthetic chemical source. 6771 came from that origin with second being biological protein antibody with 1267. Out of all these drugs the study showed that almost half were injectable (47.4%) and the next most being oral at 37.6%.

As can be seen, there are very positive things happening in the pharmaceutical R&D world currently with the number of drugs increasingly year on year. Increasing numbers of companies are getting involved in the creation of new drugs which is very important for beating and helping patients in need. To read more in detail about what has been happening please read the full article.

About the Author: Harry Kempe, a marketing intern at IIR USA, who works on various aspects of the industry including social media, marketing analysis and media. He is a recent graduate of Newcastle University who previously worked for EMAP Ltd. and WGSN as a marketing assistant on events such as the World Architecture Festival, World Retail Congress and Global Fashion Awards. He can be reached at hkempe@IIRUSA.com.

Friday, February 13, 2015

Partnerships in Clinical Trials 2015 is the world’s foremost event series for clinical development leaders and practitioners. This event draws 1100+ leaders from pharma, biotech & medical device companies, CROs, and third-party services providers in to network, learn, strategize and advance next practices in clinical trials across the globe.

*This promotion is only valid through Wednesday, February 18th at 11:59 PM. Offer cannot be applied retroactively to confirmed paying registrants or registrants that are pending payment. Cannot be combined with any other discounts or promotions. All registrants and guests are subject to IIR approval.

Regulated companies have been using formal agreements for years to define the respective quality roles, responsibilities, specifications and key performance indicators between organizations. For companies that outsource manufacturing operations, the FDA and other health authorities routinely review evidence of formal Quality Agreements during inspections. Prior to formal guidance from health authorities, many companies adopted the use of Quality Agreements as best practice, resulting in each company independently developing their own processes and templates.

The need for Quality Agreements can be traced to the ICH guidance documents Q9 Quality Risk Management (ICH Q9), which recommends supplier evaluation through audits and supplier Quality Agreements, and Q10 Pharmaceutical Quality Systems (ICH Q10), which states that the control and review of any outsourced activity are ultimately the responsibility of the sponsor. The tenets of these ICH guidance documents for industry are to build quality into processes and products (Quality by Design) rather than relying on end-product testing or inspection (Quality by Inspection).

It was not until May 2013 that the FDA issued a draft guidance, entitled Contract Manufacturing Arrangement for Drugs: Quality Agreements. In the absence of a requirement for quality agreements between study sponsors (Sponsors) and contract research organizations (CROs), we can look to this draft guidance and the ICH guidance documents.

Before creating a quality agreement, answer the following key questions:

• What is the regulatory basis for Quality Agreements and where can we find current guidance?

• What is the definition of a Quality Agreement?

• What are the essential elements of a Quality Agreement and the typical process flow between the Sponsor and the CRO or other contracted entity?

• Where does the Quality Agreement fit into the matrix of contractual agreements and the Sponsor’s supplier qualification program?

Tuesday, February 10, 2015

This general question is often asked when you meet new people for the first time and for some the answer is self-explanatory (e.g. “I teach first grade”) but for others it is more difficult. For years I heard myself saying things like “it’s hard to explain” or I’d simply give them my title (“I’m a project manager”) and wait to see if they asked more questions. I’ve even had people in my own company ask “What is it that you do exactly?”

The answer is quite long. I asked 3 of my team members to share what they say when they are asked this question and each one replied with more than a paragraph of information.

The most general description of what project managers do is that they focus on the timeline, scope and budgets of projects. They use interpersonal and leadership skills to motivate the project team and keep the team working together effectively. They prioritize tasks to determine which items are ‘must haves’ vs ‘nice-to-haves’ to keep the timeline on track. And they constantly communicate! Communication may be the most crucial skill for project managers. They must be clear and concise and must communicate on a schedule that keeps all project stakeholders on track.

Project Managers in technology-driven industries like eClinical/EDC often serve as translators between their internal technical team and external customers. They need to be skilled at gathering requirements from their customers and documenting them in a way that both the system builder and customer can understand. Additionally, our industry requires that project managers also have the ability to successfully communicate with all different types of people, with different types of needs, with different professional languages and with a wide range of technical experience - Data Managers, IT Professionals, Administrators, Statisticians, Consultants, Doctors and Nurses.

At MedNet, our iMedNet™ Software-as-a Service (SaaS) platform allows project managers to make updates directly to the eClinical websites for the studies that they manage. They are thoroughly trained in creating case report forms (CRFs) as well as configuring important business logic (such as edit checks and workflows). This requires our project managers to not only excel in all the traditional project management tasks I’ve already mentioned, but to also possess key technical skills.

In addition to directly configuring key components of eClinical solutions, project managers working in eClinical companies like MedNet use many additional technology toolsets each day. These activities can include:

It’s exciting to lead a project management team at a time where both classic and technological project management knowledge are essential to delivering superior eClinical solutions for our customers. It is clear that our industry’s constantly evolving technology solutions coupled with the latest project management skill sets will continue to be a driving force to increasing the quality and efficiency of clinical research for many years to come.

Friday, February 6, 2015

We had chance to sit down with 2015 Partnerships in Clinical Trials Event Chair Jodi Morrison, President and Chief Executive Officer, Tokai; a few years ago for a one-on-one interview. In this interview, she provides perspectives on how small and startup Pharma companies rely on partnerships to find success of their companies and emphasizes the opportunity of being small and nimble enterprises. Being so nimble, she emphasizes that employees are the key to the success of any core company.

Not only are the companies nimble, they are very connected. How can this benefit a CRO that will potentially work with CROs? With a startup Pharma company, often times the top management have spent much of their careers in the Big Pharma companies and have a huge influence within the industry. Networks are a huge value for CROs and small Pharma can often help grow them.

Thursday, February 5, 2015

Calling all industry leaders, innovators and influencers! The 24th Annual Partnerships in Clinical Trials Conference, April 22-24th, is looking to honor individual(s) working to advancing clinical research. In appreciation of your contribution, award winners will receive a Gold Pass* to attend the conference on April 22-24th valued at $3,495!

2015 Award Categories:

• Woman of the Year: A leader in the clinical trials industry, a mentor and an advocate for other women in the organization.

• Clinical Innovator of the Year: This award honors an individual or a company with a disruptive technology platform or new way to optimize clinical development that has increased the efficiency of clinical trials.

• Partnership Hall of Famer: This award honors two individuals and/or companies engaged in a partnership that moves beyond the traditional transactional model to further drug development. This award can be applied to non-traditional partnerships including academia, patient organizations, etc.

*All passes are subject approval by IIR USA and are not valid for current registrants. Woman of the Year will receive a complimentary admission to the Women's Leadership Forum & Dinner on April 22nd in lieu of a gold conference pass. Travel to the conference is not included.

Wednesday, February 4, 2015

The following tools are very useful for site monitors. Creating a site summary and subject summaries will pay off later in efficiency, accuracy and timeliness.

Site Summary
For each site, create a Site Summary that lists IRB activities, site personnel information (CVs, licenses and training), lab certificates, study drug/device receipts, notes to file, correspondence, and other documents typically found in a regulatory binder. Use this document during visits to identify and address site regulatory issues. Between visits, update the document based on communications from the site of IRB actions and other pertinent developments.

Subject Summary
Create a Subject Summary (also known as monitoring notes) for each of your sites’ study participants. This document is mainly used by the monitor to track the extent of monitoring and key data points. It can be shared with site or project management for clarification purposes.
Subject Summary contents vary by study, but usually include the activities listed in the protocol timetable. After arriving at a site, populate this document from source documents before reviewing the case report forms (CRFs). Once you begin reviewing the CRFs, highlight the data points you listed on the Subject Summary that were missed or incorrectly entered by the site that need to be queried, along with observed deficiencies that can be included in the visit report and/or post visit letter.

Use the Subject Summary to facilitate the generation of effective queries and informative findings. When writing a query, whenever possible, use your Subject Summary notes to identify the data point and corresponding source document in question so the issue is clearly presented, e.g., “The medical history included excess bleeding per the pre-op note dated 6/17/13. Please confirm to add this condition to the subject history.” Refer to the Subject Summary and attached documents when writing the Visit Report, e.g., the extent of the review during a visit and findings like adverse events, deviations, source document issues, and drug/device accountability. This tool enables a quick response to project management inquiries about a subject.

For studies that use electronic database capture (EDC), review the database entries prior to a site visit and populate the Subject Summaries. Upon arriving at a site, first review the source documentation and update the Subject Summaries, and then monitor the data in the EDC system.

Friday, January 30, 2015

The era of moving from regulatory approval to automatic worldwide adoption of a new treatment is long gone. This can be particularly true in oncology, where treatments are often expensive and can potentially be just as harmful as the disease. In the crowded oncology marketplace, collecting patient-reported outcomes (PRO) and economic data during or alongside clinical trials establishes an oncology treatment’s value to regulatory agencies, as well as to governments and private payers, maximizing your return on investment. Moreover, the rise and refinement of ePRO technologies and processes (e.g., smartphone apps, wearable devices and automated data collection/analysis) are making patient-reported data more accessible and cost-effective to collect.

Importance of PROs and Economic Endpoints in Oncology Clinical Trials
The inclusion of PROs in oncology clinical trials enables a better understanding of overall treatment effectiveness, including a more complete risk-benefit profile. In fact, numerous organizations, including the Food and Drug Administration and the American Society for Clinical Oncology, believe that PROs are essential components of clinical research in oncology.

To enable comparison of findings and ensure consistency across trials, the National Cancer Institute’s Symptom Management and Health-Related Quality of Life Steering Committee has recommended that all oncology trials include PRO measures of a core set of 12 symptoms. Using this core set simplifies the comparison of PRO findings across treatments and can facilitate the process of determining treatment value based on PRO findings. Use of additional PRO measures should also be considered to further demonstrate product value. But how do you determine the best measures? Should they be cancer-specific or based on country-specific guidance? Other factors to consider include: the goals of the clinical trial, the type/stage of cancer, existing treatment alternatives and commercial (reimbursement) targets for the product.

As the development of innovative, yet more expensive treatments in oncology continues, reimbursement agencies and other payer groups are increasingly challenged to manage budgets and provide the best possible treatment to patients. In Canada, Australia and many countries in Europe, economic evidence is required for market access and reimbursement of a new product. While such evidence is not explicitly required in the United States, health care decision makers examine treatment-associated medical resource utilization (MRU) in budgetary impact modeling and other reimbursement-related activities. Thus, as with PROs, it’s important to collect these data up front to ensure they are available for health economics analyses before market launch and for discussions with the appropriate market access agencies.

Ensure Success: Work With Health Economics and Outcomes Research Experts
Health Economics and Outcomes Research (HEOR) experts are intimately familiar with PRO-specific regulatory guidelines, as well as specific health economic, costing, modeling and other methodologies needed to satisfy diverse payer and reimbursement agency demands.

Whether HEOR expertise is available in-house or through outside consultants, it’s important to leverage proven intelligence in clinical, regulatory and marketing discussions related to oncology products, no matter the phase of development. This will ensure all PRO-related activities, including PRO selection, training and analyses, are conducted in compliance with the appropriate regulatory guidelines and follow the appropriate methodologies specific to where trials are being conducted globally. Similarly, to maximize reimbursement and product pricing success, clinical development teams should work with HEOR specialists to make sure all health economic activities are initiated early so that all necessary MRU endpoints are included in the clinical trial and the appropriate methodologies and costing techniques have been selected in advance based on the requirements for submission to appropriate agencies.

Including PROs and MRUs in oncology clinical trials should not be an afterthought. In today’s competitive health care marketplace, thoughtful planning of a clinical trial in consultation with HEOR experts can facilitate regulatory approval, lead to a PRO label claim and provide the data needed for market access and reimbursement. Incorporating PRO and MRU endpoints into a clinical trial can save you considerable time and maximize return on investment. Early preparation in consultation with experienced HEOR experts is the best plan for long-term product success.

About Decision Driver Analytics

Founded in 2006, Decision Driver Analytics provides a suite of services that covers the full range of health economics and outcomes research (HEOR) as well as the outreach materials needed to reach investors, health care providers, payers and the public. Utilizing veteran health economists, epidemiologists, biostatisticians, medical writers and reimbursement experts, DDA’s services include complete product life cycle value analysis, strategy and planning, clinical economic study services, predictive modeling analytics, definitive analysis studies, communication and sales training.Decision Driver Analytics is an Associate Sponsor at this year's Partnerships in Clinical Trials. Would you like to join them in Boston April 22-25, 2015? As a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off current rates!

Wednesday, January 28, 2015

Nobody would accuse the clinical research enterprise of being a paragon of efficient processes. It’s not that we don’t want our processes to be efficient; it’s just that the work needs to get done, no matter how inefficient the current processes are. After an initial burst of enthusiasm, process improvement projects seem to lose steam and then are quietly set aside for more pressing priorities.

The good news is that there is a proven, five-step process for improving business processes. This article will provide an introduction to the five steps, using risk-based monitoring (RBM) as an illustrative example.

But first, let’s define what we mean by “process.” In essence, a process is a standardized series of steps taken to achieve a goal. Processes simplify, streamline and regulate our activities. Formal processes are known as procedures (“standard operating procedures (SOPs)”), with well-documented steps. In our business, procedures are frequently designed to comply with governmental regulations and guidelines. Informal processes, on the other hand, just seem to exist as customary practices that are passed along by word of mouth, along with the forms and other artifacts of the process.

The Japanese method of kaizen (continuous improvement) is not limited to incremental changes. Sometimes, major changes are required to fix an obsolete or broken process. RBM, for example, requires major changes that cannot be implemented in tiny steps. Successful organizations must therefore be able to make major process improvements.

The five basic steps to significantly improve a process are: (1) map, (2) analyze, (3) redesign, (4) assign resources, and (5) implement improvements. Only after completing the first four steps can we productively move on to the fifth step, in which a process is actually improved.

Friday, January 23, 2015

The 24th Annual Partnerships in Clinical Trials Conference, April 22-24th, is looking to honor individual(s) working to advancing clinical research. In appreciation of your contribution, award winners will receive a Gold Pass* to attend the conference on April 23-24 valued at $3,495!

2015 Award Categories:

• Woman of the Year: A leader in the clinical trials industry, a mentor and an advocate for other women in the organization.

• Clinical Innovator of the Year: This award honors an individual or a company with a disruptive technology platform or new way to optimize clinical development that has increased the efficiency of clinical trials.

• Partnership Hall of Famer: This award honors two individuals and/or companies engaged in a partnership that moves beyond the traditional transactional model to further drug development. This award can be applied to non-traditional partnerships including academia, patient organizations, etc.

Register Free at www.appliedclinicaltrialsonline.com/changeEVENT OVERVIEW
Un-enrolling sites. Slow study start-up. Patient drop out or non-compliance. All of these issues--and more--have been targeted as contributors to high clinical trials costs. But what else do they have in common? They involve principal investigators and site staff. As concern grows, solutions have emerged to help physician researchers better conduct research. Other solutions are aimed to help sponsors identify issues early at the investigative site, to intervene and influence positively when necessary.

Applied Clinical Trials and Partnerships in Clinical Trials join together to bring a group of experts to discuss the importance of the investigator in clinical trials, and how to select and engage them in a true partnership.

Tuesday, January 20, 2015

Today's guest post comes from Rahlyn Gossen. She is the founder of Rebar Interactive, a clinical trial patient recruitment and digital marketing company. Rahlyn also maintains a blog, newsletter, and Twitter profile focusing on digital strategy for clinical trials.

Social media is changing the world. According to 2014 Pew Research data, about three out of four online U.S. adults use social media. Popularity varies in other countries, but overall social media adoption rates are rising worldwide. And with rising popularity comes dramatic change in consumer behaviors and expectations. No longer are people satisfied with merely being consumers of information. They also expect to share and create it. This democratization of information distribution and creation is reshaping entire industries.

Healthcare is no exception. Social media has been a key enabler in what many describe as the epatient movement. Healthcare providers are no longer the only source of healthcare information. Increasingly, patients are using social media to consume, share, and create healthcare information. And this democratization is shifting power from traditional healthcare authorities to patients. Now peer-to-peer healthcare conversations are influencing patient opinion about everything, including clinical trials.

Are you listening?

These conversations can provide key operational insights for your clinical trial. Many sponsors have shown an interest in using social media to communicate their message, particularly around patient recruitment. But listening, in this case, is at least as powerful as talking. Furthermore, listening generally carries less complexity than a social presence, particularly when it comes to regulatory issues. Sponsors who put down the bullhorn and pick up the earphones can be rewarded with valuable information, including insights into:

• Patient characteristics, attitudes, and behaviors

• Patient healthcare journey

• Patient engagement and recruitment opportunities

Listening can take many forms and vary in sophistication. For example, a simple Twitter search can be considered social media listening. Click here and scroll through some tweets. Congratulations, you have just listened to Twitter conversations about Alzheimer’s. On the more sophisticated spectrum, a variety of technology tools are available to help you collect, filter, and organize these conversations. However, even the most sophisticated technological tools require significant human analysis by someone knowledgeable about social media.

Social media listening is of course not without difficulties. For example, it can take some time to learn social media listening tools and become proficient at separating signal from noise. In addition, available listening tools are not perfect, so it’s good to have an understanding of where technology weaknesses are so you don’t inappropriately place trust in the technology. And lastly, for the data to be useful, you need someone that understands the digital and social landscape enough to contextualize it. In short, social media listening is not “plug and play.”

To learn more about the practicalities of social media listening, check out “Social Listening Intensive – Why Now, Lessons Learned, and Connections You Need” at Partnerships in Clinical Trials April 22-24 in Boston. Representatives from Shire, Novartis, and Eli Lilly will help you:

• Learn how social listening can help you understand community attitudes and behavior

• Explore the benefits and drawbacks of using social listening as a tool for patient engagement

• Gather input that may shape future research

Our ability to attract patients to clinical trials will increasingly depend on our commitment to listening to them. Social media is a valuable and accessible means to do that.

Thursday, January 15, 2015

Back in March the drug industry got a jarring demonstration of
social media’s firepower when a grassroots campaign to get a seven-year-old
cancer patient pre-approval access to an experimental anti-viral medicine
captured national attention and made headlines worldwide.

The #SaveJosh campaign—named for patient Josh Hardy, now eight—generated
sufficient pressure to force Durham, NC-based Chimerix, a small biotech, to
create a 20-patient open-label study for brincidofovir (CMX001)—in Phase 3 trials—in order to accommodate this very sick little boy.

Josh Hardy

Without question, the drug saved Josh Hardy’s life and the good news is that his condition has dramatically improved.

But the case raised some very troubling ethical questions
around “compassionate use” in an age when “who shall live” decisions can in
effect be crowdsourced via social media.

It also introduced a new dilemma for drug manufacturers in
that the advent of compassionate use crusades almost overnight and on the massive
scale facilitated by social media can potentially impede development efforts.

Kenneth Moch

According to former Chimerix CEO Kenneth Moch—who received death
threats and had to be placed under security detail during the emotionally-charged
ordeal—had the outcome been different and Josh Hardy’s response less favorable,
development of brincidofovir could’ve been delayed or even derailed,
consequently preventing the larger prospective patient population from
accessing a potentially life-saving drug.

“This
was not just about Josh Hardy; it was about the many ‘Joshes.’” - Ken Moch

“This was not just
about Josh Hardy; it was about the many ‘Joshes,’” said Moch in an interview
with Inside Clinical.

“The issue is how
to balance providing immediate access to a patient in dire need against the goal
of making a drug available to as many patients as possible by getting it
approved,” Moch emphasized.

For smaller
biotechs like Chimerix—with
just 50 employees and no other product in market—the financial strain and demands
placed on personnel by compassionate use interventions can compound the problem
by siphoning scarce resources that would’ve otherwise been dedicated to moving a
drug through the development and approval process.

“This
was the first time that social media had been used so intensely around a child
who looked and was so needy.”

“This was the
first time that social media had been used so intensely around a child who
looked and who was so needy,” Moch noted.

“The decision for making a drug available pre-approval currently rests with the company developing the drug,” he added. “And smaller companies—particularly smaller biotech companies—may not have the ability to respond to these significant social media pressures.”

It’s worth pointing out that the mainstream media—Fox News, in particular—depicted Chimerix as a Big Pharma Goliath with deep pockets, an image that served to incite public outrage and ratchet up the pressure.

Helping a gravely
ill child get access to a life-saving drug that had been denied him by a
heartless drug giant is a compelling and dangerously irresponsible narrative to spin, and the emotions it elicited at
the time appear to have made a reasoned dialogue about the complexities involved
in making compassionate use decisions impossible.

According to Moch,
Josh Hardy was one of hundreds of patients who had not been eligible to
participate in the brincidofovir SUPPRESS trial and who had petitioned Chimerix for pre-approval access but had been denied
the medication for reasons related to the company’s Phase 3 development
efforts.

Social and
mainstream media pressure enabled Josh Hardy to get the drug and to survive,
but the event set a troubling precedent by relegating the decision to the whims
of public opinion and inequitable influence.

How
do you decide who can and cannot have access to an experimental drug? Contacts?
Money? Social media?

“How do you decide
who can and cannot have access to an experimental drug? Is it through social
media? Is it through contacts? Is it through money? Is that fair and equitable?
I think not,” said Moch.

The Josh Hardy case has forced the industry and regulators to
scrutinize expanded access to experimental drugs. BIO and PhRMA have since responded and the FDA has formed a task force.

But Moch emphasized that
companies must be prepared to address social media campaigns for pre-approval
access to drugs they’re developing head on or face potentially disastrous consequences
to their development efforts.

In this podcast for Inside Clinical—the official interview series of
the 24th Annual Partnerships in Clinical Trials conference—Ken Moch
discusses the issues around “compassionate use” and what manufacturers can
learn from his experience.

ABOUT THE AUTHOR/INTERVIEWER
Marc Dresner is IIR USA's senior editor and special communication projects lead. He is the former executive editor of Pharma Market Research Report, a confidential newsletter for marketing researchers in the pharmaceutical industry. He may be reached at mdresner@iirusa.com. Follow him @mdrezz.

Wednesday, January 14, 2015

Partnerships in Clinical Trials 2015 is the world’s foremost event series for clinical development leaders and practitioners. This event draws 1100+ leaders from pharma, biotech & medical device companies, CROs, and third-party services providers in to network, learn, strategize and advance next practices in clinical trials across the globe.

Tuesday, January 13, 2015

Yes according to Joseph V. Gulfo, MD, MBA, in a recent post at The Hill. According to the article, 41 new drugs were approved in 2014 and 40% of them were orphan drugs. The Orphan Drug act is a big part of this - pushing exclusivity and driving profits. Gulfo sees this as a problem - because they see drug approvals but the public continues to fewer drug approvals that drive health. Instead those being approved and their high costs are seeing profits that lean on blockbuster drug levels.

So to conclude his article, Gulfo points out what the Orphan Drug Act has taken away from - science and medicine are no longer driving development for health. Instead it's the policies of that the FDA set forward for drug approvals. Do you agree?

Dr. Gulfo will be joining us at Partnerships in Clinical Trials on Thursday, April 23 for the presentation Picking up the Pieces of Clinical Trial Failure: How I Battled the FDA and Won. For more information on this session and the rest of the program, download the agenda. If you'd like to join us, as a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off current rates - lowest rates of the year expire Friday, January 16.

Monday, January 12, 2015

Calling all non-profit patient representatives and organizations! Leading with the patient voice a top priority for the 24th Annual Partnerships in Clinical Trials Conference (April 22-24th in Boston, MA) which is why we're looking for representatives to be a part of our event community. New for 2015, patient organizations and representatives will have a home base in our "Patient Pavilion". Qualifying organizations will receive 2 conference exhibit hall passes and a table top stand.

About the Conference: Partnerships in Clinical Trials is the world’s foremost event series for clinical development leaders and practitioners. This event draws 1100+ leaders from pharma, biotech & medical device companies, CROs, and third-party services providers in to network, learn, strategize and advance next practices in clinical trials across the globe.

Interested in participating or know a Patient Organization who would be? Please email Program Director, Marina Adamsky. Hurry, spots are limited and filling up fast!

Friday, January 9, 2015

The Institute for International Research is looking for speakers from sponsor companies for our upcoming Partnering with Central Labs Conference to be held in conjunction with the 24th Annual Partnerships in Clinical Trials Conference -- April 21-22, 2015 in Boston, MA.

DEADLINE EXTENDED! We are currently recruiting pharmaceutical/biotech/medical device professionals in the procurement, outsourcing, relationship management with Central/ECG/Imaging relationship management, quality and category leads. Plus, Companion Diagnostics Program Directors and Heads of Translational Medicine.

Topics include:

• Innovation in Microsampling

• Strategies to Get to First Patient In and Site Initiation Quicker

• Interpret Testing for Drug Induced Liver Injury

• Managing Ancillary Vendors at a Small to Midsized Pharma Company

• Delve into the Shipping Costs Between Labs + Shipping Companies

• Examining the Regulatory Requirements for a Successful Submission for CDx

• The Development of Regulated Next-Generation Sequencing-Based Clinical Trial Assays and CDx

• Lab Selection and Qualification Criteria

• Use of a Local vs. Global Lab in Complex Clinical Trials

• New Developments in Biomarker Testing

• Sponsor/CRO/Lab Relationships – Using Metrics to Benchmark Success

• Best Practices in Lab Data Integration

• Contracting with Central Labs - What to Put in Your Contract

We invite you to submit a proposal* for a speaking opportunity directly to Marina Adamsky.

Thank you and we look forward to welcoming you to Boston in April!

*Note: Proposals are subject to review IIR to ensure the overall quality of the conference program. Please note that due to limited speaking slots, preference is given to abstracts from those within pharmaceutical and biotech companies, regulators, and those from academic centers. A select number of suppliers providing services to the industry are offered presentation opportunities in conjunction with an event sponsorship. Please email Patricia Rose at prose@IIRUSA.com.

Thursday, January 8, 2015

We invite clinical trial industry professionals with valuable knowledge and insights to participate in Avoca's 2015 Annual Industry Survey. The topic of this year's survey is Innovative Approaches to Clinical Development.

For more than 10 years, The Avoca Group has surveyed industry executives and managers to gain a better understanding of key trends affecting the conduct of outsourced clinical development. The insights gained from this research have proven valuable to industry leaders who are working to strengthen relationships and enhance R&D productivity. Your participation is instrumental in facilitating these improvements.

In addition to receiving an Executive Summary of key findings from our research, upon completion of the survey you will be entered into a drawing for one of two Amazon gift cards valued at US $500. As always, your personal information will remain confidential and will not be associated with your survey responses in any manner.

Tuesday, December 23, 2014

It doesn’t show signs of stopping…
Let is sale! Let it sale! Let it sale!

‘Tis the season! As our holiday gift to you, here’s 30% off the standard rates when you register for Pharma and Healthcare events from now through Wednesday, December 31! Use the code “Holiday30” at checkout.

Monday, December 22, 2014

Rick Morrison, Founder and CEO of Comprehend Systems recently took and in-depth look at some of the major considerations an company should look at before they venture into low-cost countries for clinical trials. With the steep cost of clinical trials - Astra Zeneca faced a cost of $11.7 billion for each of the five drugs it brought to market from 2007-2011 - it's important that companies find efficient, low cost and ethical ways to bring drugs to market faster and safer.

Morrison suggests looking into expanding clinical trials beyond domestic trials to reap the benefits of diverse patient populations, the shrinking of cost and time to market for successful drugs and the ability to test the products in the countries and patient populations to which they'll be sold. Not only are these benefits clear within themselves but the number of patients presented in a global clinical trial allows to companies to bring drugs to market an average of 6-7 months faster.

What are some of the benefits you see from expanding clinical trials beyond the traditional borders?

This April at Partnership in Clinical Trials, we will host Effects of Globalization on Clinical Development a full day workshop to kick off the 2015 event. Presentations will look at global data, streamlining outsourcing, patient recruitment and more. For more information on this session and the rest of the program, download the agenda. As a reader of this blog, when you register to join us and mention code XP2000BL, you can save $100 off the current rate. We're also giving away a few free passes on Twitter. Find out how you can win.

Friday, December 19, 2014

Innovation in the life science industry is evolving. With over 90% of innovations occuring outside of big Pharma's research and development labs, it's important that big pharma be in tuned with what's going on in and leverage the expertise across the value chain. In a recent interview at partnering 360®, Jochen Maas, the General Manager of Research and Development at Sanofi Germany sat down and shared his thoughts on capitalizing on external innovation with partners and identifying the real experts in the Pharma and Biotech industry to get products to the patients as fast as possible.

Watch the interview here:

Partnering with experts in the field a company trying to excel in - especially Pharma and Biotech - is critical. What do you see are the benefits of working with the innovative biotech industry for clinical trials?

Wednesday, December 17, 2014

Today's guest post comes from Rahlyn Gossen. She is the founder of Rebar Interactive, a clinical trial patient recruitment and digital marketing company. Rahlyn also maintains a blog, newsletter, and Twitter profile focusing on digital strategy for clinical trials.

“Futurist, pharma tycoon, satellite entrepreneur, philosopher. Martine Rothblatt, the highest-paid female executive in America, was born male. But that is far from the thing that defines her. Just ask her wife. Then ask the robot version of her wife.”

While reading the introduction of an online New York magazine profile of Martine Rothblatt, I wondered how accurately it reflected the content of the full article. As someone who reads a lot on the Internet, I’ve come into contact with my share of sensationalistic and hyperbolic hooks. But by that point my curiosity was piqued, so I continued reading. And the article did not disappoint. Martine Rothblatt is every bit as fascinating a person as the snippet above would lead you to believe.

In the 1990s Martine’s youngest child was diagnosed with primary pulmonary hypertension, a rare and fatal disease. Martine took action. She started the PPH Foundation, educated herself about pulmonary conditions, and founded a pharmaceutical company. Her company, United Therapeutics, went public in 1999 and received approval for a PPH drug last year. Martine’s daughter, whose condition inspired her foray into pharma, turns 30 this year and works for United Therapeutics.

Prior to founding United Therapeutics, Martine was already quite accomplished and even considering retirement. She had founded Sirius Radio and took it public in 1994. Prior to Sirius, Martine founded GeoStar, a GPS-based navigation system. Martine began her career as a communication satellite lawyer after earning a combined law and MBA degree from UCLA.

Currently, Martine is expanding United Therapeutics and promoting a new book titled Virtually Human: The Promise—And Peril—of Digital Immortality. Martine considers herself a transhumanist, which according to the article, is “…a particular kind of futurist who believes that technology can liberate humans from the limits of their biology—including infertility, disease, and decay, but also, incredibly, death.” According to Martine, artificial intelligence will be the primary vehicle to enable this new future, where even the dead can be reanimated as digital beings.

Thursday, December 11, 2014

COPD is a disease that is quickly growing to be one of the biggest expenditures in healthcare. According to the NIH, it's predicted that by 2020, the disease will cost the United States $49 billion. In 2010, it cost the state of Florida $2.5 billion.

There is no cure, and the UK's NIHR points out that the only preventable cure for this disease is to quit smoking. Over 35,000 patients have participated in research and between 2008 and 2009, there were 157 new studies that began. See World Startup Report's research here.